abstract |
The present invention relates to iRNAs, such as double-stranded ribonucleic acids (dsRNAs), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene and the complement component C9 gene, and compositions of CFB, C9 and/or C3 Such iRNAs, e.g., dsRNAs, for inhibiting expression and for treating subjects with complement component-associated diseases such as paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome , to a method of using the composition. |